On December 15, 2021 Nuvation Bio Inc. (NYSE: NUVB), a biopharmaceutical company tackling some of the greatest unmet needs in oncology by developing differentiated and novel therapeutic candidates, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to NUV-422, a cyclin-dependent kinase (CDK) 2/4/6 inhibitor, for the treatment of patients with high-grade gliomas, including glioblastoma multiforme (Press release, Nuvation Bio, DEC 15, 2021, View Source [SID1234597256]). NUV-422 received Orphan Drug Designation for the treatment of patients with malignant gliomas from the FDA in the first quarter of 2021.

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"We are pleased that NUV-422 received FDA Fast Track designation because it highlights the serious unmet need of patients with brain cancer and the potential of our lead drug candidate NUV-422 to serve as an innovative new treatment option for high-grade gliomas, including glioblastoma multiforme," said David Hung, M.D., founder, president, and chief executive officer of Nuvation Bio. "Enrollment is ongoing in our expanded Phase 1/2 monotherapy study of NUV-422 for the treatment of adults with recurrent or refractory high-grade gliomas and solid tumors. We look forward to continuing to work closely with the FDA to expedite the development of NUV-422 with data from the Phase 1 portion of the study, which is on track for 2022."

NUV-422 is the Company’s lead investigational CDK2/4/6 inhibitor program that works to overcome the limitations of CDK4/6 inhibitors. CDK4/6 inhibitors are known clinical entities with proven efficacy, but some cancer cells can evade these treatments by increasing signaling through CDK2. Inhibition of CDK2 in addition to CDK4/6 cuts off the tumor’s natural escape route. NUV-422 is a potent inhibitor of CDK 2, 4, and 6. Preclinical studies have shown that NUV-422 has favorable blood-brain barrier penetration. NUV-422 is also designed to limit CDK1 inhibition, a potential cause of toxicity in other second-generation inhibitors. In addition to high grade gliomas, NUV-422 is currently being studied in HR+ HER2- advanced breast cancer (with and without brain metastases) and metastatic castration resistant prostate cancer (mCRPC). Fast Track designation is designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need with the goal of getting important new drugs to patients earlier. A drug that receives Fast Track designation is eligible for more frequent meetings and communications with the FDA, accelerated approval and priority review if certain criteria are met, and more.

On December 15, 2021 Citius Pharmaceuticals, Inc. ("Citius" or the "Company") (Nasdaq: CTXR), a late-stage biopharmaceutical company dedicated to the development and commercialization of first-in-class critical care products with a focus on oncology, anti-infective products in adjunct cancer care, unique prescription products, and stem cell therapies, reported business and financial results for the fiscal full year ended September 30, 2021 (Press release, Citius Pharmaceuticals, DEC 15, 2021, View Source [SID1234597255]).

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Fiscal Full Year 2021 Business Highlights and Subsequent Developments

Completed subject treatment in December 2021 in Pivotal Phase 3 trial of I/ONTAK (E7777) with topline results anticipated in the first half of 2022 and a biologics license application (BLA) submission in the second half of 2022;
Acquired Dr. Reddy’s Laboratories’ license for late-Phase 3 oncology immunotherapy I/ONTAK for the treatment of cutaneous T-cell lymphoma (CTCL) and other cancer indications;
Received third positive recommendation from independent Data Monitoring Committee to continue the Mino-Lok Phase 3 clinical superiority trial as planned without modifications; no safety concerns identified;
Completed dose-ranging proof-of-concept sheep study of iPSC-derived novel induced-mesenchymal stem cells (i-MSCs) for the treatment of acute respiratory distress syndrome (ARDS); analysis and documentation underway for peer-reviewed publication submission;
Expanded clinical, manufacturing and commercial capabilities with the addition of seasoned pharmaceutical executives;
Citius added to the Russell 3000 and 2000 indexes; and,
Raised net proceeds of $120.6 million in financing activities during the year.
Financial Highlights

Cash and cash equivalents of $70.1 million as of September 30, 2021;
R&D expenses were $12.2 million for the full year ended September 30, 2021, compared to $8.8 million for the full year ended September 30, 2020;
G&A expenses were $9.8 million for the full year ended September 30, 2021, compared to $8.1 million for the full year ended September 30, 2020;
Stock-based compensation expense was $1.5 million for the full year ended September 30, 2021, compared to $0.8 million for the full year ended September 30, 2020; and,
Net loss was $23.1 million, or ($0.23) per share for the full year ended September 30, compared to a net loss of $17.5 million, or ($0.45) per share for the full year ended September 30, 2020.
"2021 was a transformative year for Citius as we positioned the company financially and strategically to drive growth. We raised more than $120 million in proceeds to support our activities, providing us with the flexibility to advance our clinical programs and invest in opportunities for additional growth. With the recent addition of cancer immunotherapy I/ONTAK to our portfolio, we now have a robust pipeline that includes two late Phase 3 programs and three potentially first-in-class products," stated Myron Holubiak, President and Chief Executive Officer of Citius Pharmaceuticals.

"As we move into 2022, we anticipate multiple positive milestones. These include: accelerated enrollment and completion of the Mino-Lok trial during the year, topline results in the first half of 2022 for the recently completed I/ONTAK Pivotal Phase 3 trial followed by a BLA submission in the second half of the year, initiation of the Halo-Lido study in early 2022 and completion of the study by the end of the year, and continued progress on Mino-Wrap and our iPSC-derived mesenchymal stem cells for the treatment of acute respiratory distress syndrome (ARDS). With the addition of key personnel to our clinical, manufacturing and commercial teams, we are aligning our resources to ensure continued progress across each of our development programs, and the successful launch of potentially two commercial products in 2023. We believe our strong balance sheet will allow us to execute our development programs as planned in 2022 and we do not anticipate a need to raise additional capital in the coming year," concluded Mr. Holubiak.

Full Year 2021 Financial Results:

Liquidity

As of September 30, 2021, the Company had $70.1 million in cash and cash equivalents. During the fiscal year ended September 30, 2021, the Company received net proceeds of $120.6 million from financing activities.

In January 2021, the Company closed a private placement for common stock and warrants totaling gross proceeds of approximately $20 million and net proceeds of $18.5 million. In February 2021, the Company closed a registered direct offering of its common stock and warrants for gross proceeds of $76.5 million and net proceeds of $71 million. During the year ended September 30, 2021, the Company received $31.2 million in proceeds from the exercise of common stock options and warrants.

On June 21, 2021, stockholders approved an amendment to the Company’s Articles of Incorporation to increase the authorized number of shares from 210,000,000 to 410,000,000 and the authorized number of common shares from 200,000,000 to 400,000,000. As of September 30, 2021, the Company had 145,979,429 common shares issued and outstanding.

In 2021, the Company raised a total of $127.6 million through financing activities. We estimate that we will have sufficient funds for our operations through March 2023.

Research and Development (R&D) Expenses

R&D expenses were $12.2 million for the full year ended September 30, 2021, compared to $8.8 million for the full year ended September 30, 2020. The increase of $3.4 million is primarily due to an increase in research and development expenses for our proposed novel cellular therapy for ARDS of $6.1 million, of which $5 million was a license fee paid to Novellus, and an increase in R&D expenses related to the I/ONTAK license and Mino-Wrap, offset by decreases in research and development expenses related to our Mino-Lok and Halo-Lido product candidates.

We expect that research and development expenses will increase in fiscal 2022 as we continue to focus on our Phase 3 trial for Mino-Lok, progress the Halo-Lido product candidate, and continue our research and development efforts related to ARDS, Mino-Wrap and I/ONTAK (E7777).

General and Administrative (G&A) Expenses

G&A expenses were $9.8 million for the full year ended September 30, 2021, compared to $8.1 million for the full year ended September 30, 2020. The primary reason for the increase is costs associated with additional compensation costs for new employees and performance bonuses. General and administrative expenses consist primarily of compensation costs, professional fees related to our capital raising activities, corporate development services, and investor relations.

Stock-based Compensation Expense

For the full year ended September 30, 2021, stock-based compensation expense was $1.5 million as compared to $0.8 million for the prior year. The increase reflects expenses related to new grants made by Citius and the NoveCite stock option plan.

Net loss

Net loss was $23.1 million, or ($0.23) per share for the year ended September 30, compared to a net loss of $17.5 million, or ($0.45) per share for the year ended September 30, 2020. The increase in net loss is primarily due to the $3.4 million increase in our research and development expenses and a $1.6 million increase in general and administrative expenses.

On December 15, 2021 Rakuten Medical, Inc reported that the U.S. Food and Drug Administration (FDA) has accepted the company’s Investigational New Drug (IND) application to begin clinical studies of RM-1995 photoimmunotherapy in patients with advanced cutaneous squamous cell carcinoma or with head and neck squamous cell carcinoma (Press release, Rakuten Medical, DEC 15, 2021, View Source [SID1234597253]).

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RM-1995 photoimmunotherapy treatment is an investigational drug-device combination being developed by Rakuten Medical, Inc. The drug portion of the treatment, RM-1995, is a conjugate of a photoactivatable dye (IRDye 700DX [IR700]) and a monoclonal antibody specific for cell-surface interleukin 2 (IL-2) receptor α-chain (CD25). The device portion of the treatment is a laser device system (PIT690 Laser System) used to activate IR700 by illumination with 690nm nonthermal red light. Tumor-resident regulatory T cells (Tregs), that inhibit antitumor immune responses to promote tumor growth, can be specifically targeted with anti-CD25 antibodies1. Rakuten Medical, Inc is investigating RM-1995 as a potential new treatment to provide targeted, direct reduction of Tregs specifically within light-treated tumor lesions to induce systemic anticancer immune responses.

"We are very excited to have achieved this regulatory milestone and delighted to be moving RM-1995 into the clinic," said Mickey Mikitani, Chief Executive Officer of Rakuten Medical, Inc. "RM-1995 has the potential to attack the cancer in a novel way, targeting Tregs. Based on data from preclinical studies, RM-1995 photoimmunotherapy treatment is expected to result in acute killing of Tregs within the tumor. We are developing drugs using various antibodies in our drug discovery program based on Rakuten Medical’s IlluminoxTM platform, and RM-1995 is the second pipeline drug developed on this platform. We will continue to advance our business with a sense of speed so that we can deliver these important treatments to as many patients as possible."

On December 15, 2021 Syndax Pharmaceuticals, Inc. ("Syndax," the "Company" or "we") (Nasdaq: SNDX), a clinical-stage biopharmaceutical company developing an innovative pipeline of cancer therapies, reported the pricing of an underwritten public offering of 3,157,144 shares of its common stock, and to certain investors pre-funded warrants to purchase 1,142,856 shares of its common stock at an exercise price of $0.0001 (Press release, Syndax, DEC 15, 2021, View Source [SID1234597251]). The public offering price of each share of common stock is $17.50 and the public offering price of each pre-funded warrant is $17.4999 per underlying share, which represents the per share public offering price for the common stock less the $0.0001 per share exercise price for each such pre-funded warrant. The aggregate gross proceeds from this offering are expected to be approximately $75.3 million, before deducting underwriting discounts and commissions and other offering expenses payable by Syndax. In addition, Syndax granted the underwriters a 30-day option to purchase up to an additional 645,000 shares of common stock. All of the shares of common stock and pre-funded warrants are being sold by Syndax. The offering is expected to close on December 20, 2021, subject to customary closing conditions.

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Goldman Sachs & Co. LLC and Cowen are acting as joint book-running managers for the offering. BTIG is acting as lead manager for the offering. B. Riley Securities is acting as co-manager for the offering.

The shares are being offered pursuant to a "shelf" registration statement previously filed and declared effective by the Securities and Exchange Commission (SEC). A preliminary prospectus supplement and accompanying prospectus relating to the offering will be filed with the SEC and will be available on the website of the SEC at www.sec.gov. When available, copies of the preliminary prospectus supplement, final prospectus supplement and accompanying prospectus relating to the offering may be obtained from: Goldman Sachs and Co. LLC, Attention: Prospectus Department, 200 West Street, New York, NY 10282, telephone: 866-471-2526, facsimile: 212-902-9316 or by emailing [email protected]; or Cowen and Company, LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, New York 11717, or by email at [email protected], or by phone at (833) 297-2926.

This press release shall not constitute an offer to sell, or the solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction. Any offer, if at all, will be made only by means of a prospectus supplement and accompanying prospectus, which are a part of the effective registration statement.

On December 15, 2021 Inventia Life Science, a world leader in advanced 3D cell cultures for research and clinical purposes, has reported the close of a US $25M Series B funding round, led by Blackbird Ventures (Press release, Inventia Life Science, DEC 15, 2021, View Source [SID1234597250]). Inventia has also announced the launch of its US operations with the appointment of Dwayne Dexter as its Director of US Sales. Through this Series B round of financing, Inventia plans to market its RASTRUM 3D cell culture platform worldwide and grow its team from 36 to 150 employees by the end of 2024. In particular, it aims to develop a strong presence in the US, where the biomedical research and drug discovery markets are currently estimated to be worth more than US $40 billion. This Series B brings Inventia’s total funding to date to US $32 million. The round also included significant re-investment from Skip Capital.

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"This new round of financing is a very significant milestone for Inventia. The funds will enable us to scale up and take full advantage of the increasing global interest in such things as new approaches to cancer research, and the development and validation of new drugs," says Inventia’s Founder and CEO, Dr. Julio Ribeiro. "The biomedical applications for the use of the RASTRUM platform are very diverse. For example, it can be of enormous benefit to the pharma industry, by allowing new drugs to be tested in a 3D cellular environment and eliminated if necessary, long before they reach the stage of clinical trials. This can reduce by hundreds of millions of dollars the total cost of bringing a successful drug to market. Similarly, cancer researchers can now work at scale with cell models that mimic the human body almost exactly and produce research results that are more accurate and predictive than before."

Alongside this Series B round of funding, Inventia has also appointed Dr. Dwayne Dexter as its Director of US Sales. Dwayne will be instrumental in growing the pharmaceutical and academic customer base and driving adoption of Inventia’s technology in the US and Canada. Currently, three of the leading US pharmaceutical companies are Inventia customers. Prior to joining Inventia, Dwayne served as the Director of US Operations at Mimetas, a US-based organ-on-a-chip provider.

Niki Scevak, Partner at Blackbird Ventures, commented on the news, saying, "We are thrilled to help accelerate the adoption globally of Inventia’s unique 3D cell culture platform. It’s a technology that improves cancer and other cellular research in a fundamental way, and the benefits of this will reshape many biomedical industries, in particular the pharmaceutical industry," said Scevak. "Our financial support of Inventia reflects our firm belief that there is a broad-based and urgent need in the biomedical world for what the company has developed and that its impact is going to be truly generational."

Kim Jackson, Founder at Skip Capital, commented, "Inventia has phenomenal founders who are building a high growth biotech company. I believe their technology will revolutionize the way that pioneering researchers in areas such as cancer discover and test new and innovative drugs."